Libmerdi, a €2.5 million treatment for a rare disease, is now available in Ireland, Belgium and the Netherlands.
advertisementHealth regulators in Ireland, Belgium and the Netherlands have approved the breakthrough gene therapy LibMerdi, making it the most expensive drug to receive official approval in European countries.
Irish Health Minister Stephen Donnelly announced that their respective countries’ health services have approved the gene therapy Atidarsurgen Autotemsel (also known as RibMerdy), developed by Orchard Therapeutics. did.
The treatment, which costs around 2.5 million euros per dose, is aimed at the most severe cases of metachromatic leukodystrophy (MLD), a rare genetic disease that causes damage to nerve cells.
Under the Beneluxa concept, the three countries, Luxembourg and Austria, negotiated directly with manufacturers, and each country negotiated price reductions.
The final cost has not been disclosed, but estimates of the savings range from 25% to up to around 65% in some cases, according to Irish broadcaster RTE.
“By working together across borders, we can bring vital medicines to patients. To improve their lives and save lives,” said Belgian Minister of Health and Social Affairs Frank Vandenbroucke. stated in a statement.
“That’s why we need to cooperate more in Europe. Beneluxa is a great example of cooperation, which allows pharmaceutical companies to avoid fighting between countries,” he added.
Rare disease with poor prognosis
In Europe and North America, MLD affects 1 in 40,000 to 1 in 100,000 people.
In the infantile form, progression to death usually occurs within 5 to 6 years, whereas in the juvenile form, it occurs 10 to 20 years after onset.
According to , Ribmerdi is used in children from infancy to early childhood with this disease. European Medicines Agency (EMA).
It is a one-time administration intended to provide a permanent solution.
This treatment involves extracting a patient’s own stem cells from bone marrow or blood, modifying those stem cells to express the correct genes, and reintroducing them into the patient’s bloodstream.
Why is it so expensive?
Gene therapy involves modifying a patient’s genes to correct or alleviate inherited diseases and requires specialized techniques and advanced equipment.
The development and production of these treatments is a resource-intensive process.
Libmeldy, for example, took about 20 years to develop, with a 10-year gap between first human clinical trials and approval by the EMA in 2020. BBC.
This requires gene editing tools, viral vectors for gene transfer, and cell manipulation equipment.
advertisementAccording to Spanish media, the United States also has laws that favor big drug companies, and although European countries have the ability to negotiate prices, costs could be affected by a less regulated market. el pais.